New York-based biotech Synaptogenix revealed disappointing data from a phase 2 clinical trial of its protein kinase C activator for the treatment of advanced Alzheimer's disease this week, announcing that the trial had failed to meet its primary endpoint.
The randomized, double-blind, placebo-controlled study compared Bryostatin-1 to placebo for long-term efficacy in the treatment of advanced and moderately severe AD in the absence of memantine. The study’s primary endpoint was for the drug to improve patients’ Severe Impairment Battery (SIB) following completion of the second course of treatment, given 28 weeks after the first dosage. However, the biotech shared that the average increase in SIB total score for the Bryostatin-1 group was 1.4 points, while the placebo cohort reported an increase of 0.6 points.
Bryostatin-1 works by binding and inhibiting the cell-signaling enzyme protein called kinase C, which can have therapeutic effects such as inducing cell apoptosis and helping anticancer drugs to work better. Researchers have found that the underlying pharmacological mechanisms for central nervous disorders involve the induction of synthesis of proteins required for long-term memory and a reduction of neurotoxic accumulation of amyloid plaques and tau proteins. Synaptogenix has been evaluating the drug potential for other disorders such as the rare disease Fragile X syndrome, for which the FDA granted it Orphan Drug designation.
Even though the drug is Synaptogenix’s lead asset, the company said in its recent statement that it “remains well-funded with approximately $38.5 million in cash as of December 15, 2022." As for next steps, the biotech said it will be conducting a full review of the trial data to determine future plans.
